Figure 4.

Antisense oligonucleotides (AONs) tested in immortalized cells for exon-skipping pre-clinical studies. (A) Validation of AO51 efficacy for exon 51 skipping on primary (Prim.; Duchenne muscular dystrophy (DMD)) and immortalized cultures of a Δ48-50 patient with DMD. (B) Evaluation of the efficacy of various AONs on control immortalized cells targeting exons 17 and 18.